F. Stephen Hodi, M.D., of the Dana-Farber Cancer Institute, Boston, and colleagues conducted a phase 2 clinical trial in which 245 patients with unresectable (unable to be removed by surgery) stage Ill or IV melanoma were randomly assigned to receive ipilimumab (intravenously) on day 1 plus sargramostim (injected beneath the skin) on days 1 to 14 of a 21-day cycle (n = 123) or ipilimumab alone (n = 122). The researchers compared the effect of these treatments on length of overall survival, progression-free survival, response rate, safety, and tolerability.

Median follow-up was 13.3 months. The overall survival was significantly improved with the addition of sargramostim to ipilimumab. The median overall survival was 17.5 months for the ipilimumab plus sargramostim group and 12.7 months for the ipilimumab-only group. The 1-year overall survival was 68.9 percent for the combination treatment group and 52.9 percent for the ipilimumab-only group. There was no difference in progression-free survival. Adverse events were more common in the ipilimumab-only group. Toxicity was significantly lower in the ipilimumab plus sargramostim group than in the ipilimumab­only group.